Present and future of disease-modifying therapies in multiple system atrophy

Abstract

Through the last decade seven clinical trials on Multiple System Atrophy have been published, virtually all of them reported negative results. Patients and family remain hopeful while facing this devastating disease, but as doctors we still cannot offer them disease-modifying therapies. The field has seen many advances regarding pathophysiology, translational research, diagnostic accuracy, natural history and imaging, but successful treatment remains elusive. This review provides an overview of the available tools for designing clinical trials, critically analyzes the past studies and describes the knowledge obtained from them, and finally gives some orientation for future trials that could meet the current needs of patients and clinicians, overcoming the hurdles met by previous studies.

Keywords: Alpha-Synuclein; Clinical trials; Disease modifying therapies; Treatment.